Natural History and Treatment Outcomes of Congenital Thrombotic Thrombocytopenic Purpura: A Retrospective Longitudinal Cohort Study
Abstract
Background: Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra-rare, life-threatening thrombotic disorder. The real-world clinical trajectory and management of cTTP are not well defined.
Objectives: Describe patient characteristics, quantify the incidence and prevalence of clinical manifestations and disease-related complications, and describe treatment patterns and treatment-related outcomes of patients with cTTP.
Methods: In this retrospective, multinational, longitudinal cohort study, data were abstracted from the medical records of patients with severe hereditary ADAMTS13 deficiency (<10% activity) at 9 participating sites across Europe and the United States. Eligible patients experienced an index event (cTTP diagnosis, an acute TTP event or TTP manifestation, a cTTP-related clinical event, or prophylaxis) between January 1, 2009, and December 31, 2017. Data collection was from January 1, 2009, to December 31, 2020. A post hoc analysis was conducted among patients who matched inclusion criteria for a phase 3 trial (NCT03393975), and who received plasma-based therapy (PBT) prophylaxis at least once per month, up to 3 times per week.
Results: Medical records from 78 patients (61 [78.2%] female) were included in the study. The mean (SD) follow-up was 8.1 (3.1) years. Ninety-two acute TTP events were recorded in 55 (70.5%) patients (overall event rate, 0.145 events per person-year). Eighty (87.0%) acute TTP events occurred in the absence of prophylactic…
How strong is the evidence?
Evidence Hub reads this as a cohort study, done in people. Its evidence level is 3 of 5 (Moderate · trial / cohort / secondary analysis). Trials without a large randomized design, cohorts and secondary analyses give moderate evidence. Reported size: 78 participants or records.
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